When all else failed, the promise of corporate profits for pediatric cancer drugs did what cajoling to save children could not.

Legislation by Texas Rep. Michael McCaul, signed into law Monday by President Barack Obama, offers drug companies multimillion-dollar incentives to pioneer medications for rare childhood diseases that afflict too few kids to make a profit.

The legislation is meant to remedy a chronic mismatch in which the FDA has approved dozens of new drugs to combat adult cancers since 1980 – and only one for the treatment of childhood cancer.

“We’re giving companies incentives to make money because the free market has failed to develop these medications,” says McCaul, a five-term Austin Republican and father of five who founded the 94-member Congressional Childhood Cancer Caucus.

The measure “fundamentally transforms the way drug companies look at rare pediatric diseases and compensates for market failures that have prevented any new treatment for pediatric cancer from being developed in a generation,” he added.

White House Press Secretary Jay Carney said the legislation would increase the ability of the FDA to “provide timely and expedited review and approval of applications for prescription drugs and medical devices” as well as modify FDA authority so the agency could offer incentives for manufacturers “drugs intended for use by children.”

Dr. Eugenie Kleinerman, head of pediatrics at the University of Texas M.D. Anderson Cancer Center, said clinicians have been frustrated for years by the absence of state-of-the-art anti-cancer medications tailored to children. M.D. Anderson treats 2,000 pediatric patients a year including 200 in clinical trials.

“But this is absolutely a move forward,” says Kleinerman. “It will shine a light on the fact that we really haven’t had access to the latest new therapies.”

Development of such drugs still will take five to seven years.

Some 12,500 children up to the age of 18 are diagnosed with cancer each year – a fraction of the hundreds of thousands of adults who confront cancer diagnoses each year.

Helping other families

Families hit by childhood cancer embraced the promise of tailor-made drugs even though it is too late for their own kids.

“There’s been no excuse for having no drugs to treat children because there’s more money to be made treating adults,” says Donna Culliver, of Brenham, whose 4-year-old son, Adam, died in 2003 from fast-moving acute myelogenous leukemia.

She and her husband started Adam’s Angels Ministry to help families and began lobbying for ways to encourage drug companies to do more.

“We can’t help our son, but we can help other families that receive a diagnosis of childhood cancer,” Culliver said. “To us this legislation is a huge blessing – Adam’s death wasn’t in vain, it was for a reason.”

In return for developing drugs for the small and unprofitable market of rare childhood diseases, cooperating pharmaceutical manufacturers could earn vouchers for faster Food and Drug Administration approval of new and potentially more profitable drugs.

By cutting FDA review time by 40 percent from an average of 10 months to six months, participating drug manufacturers could reap rewards of up to $500 million in additional sales on patent-protected medications that reach the competitive marketplace first, Congress estimates.

Drug companies also could sell the vouchers to other companies jockeying with rivals to get their drug to market first.

The legislation provides for three vouchers to be issued in coming years, followed by an audit on the effectiveness of the approach by Congress’ watchdog Government Accountability Office.

Drug companies

Kate Connors, spokeswoman for the Pharmaceutical Research and Manufacturers of America, said drug manufactures poured almost $50 billion into developing and discovering new drugs last year and will support any provisions that “encourage research into how better to treat children.”

However, she added, the industry organization had not assessed yet the incentives included in the new law.

Advocacy organizations and physicians say they will be reaching out to drug manufacturers in hopes of piquing interest and speeding development of medications targeting rare childhood diseases.

“This will be a sea change in drug development,” forecasts Nancy Goodman, who founded Kids v. Cancer after the death of her son Jacob at the age of 10 in 2009 from a pediatric brain cancer.

“We will work with companies to help shepherd them through the FDA review process,” said Goodman, who worked with doctors and researchers at more than 35 institutions across a dozen countries in an effort to find a medication to save her son’s life.

“We do so much up here on Capitol Hill that shows we’re kind of spinning our wheels on things that never get done,” said McCaul. “This is refreshing – it will make a real difference in the lives of children.”

McCaul’s wife, Linda Mays McCaul, a multimillionaire heiress to her family’s Clear Channel Communications, is a member of the board of M.D. Anderson.